Broad Institute, Boston Children's, and Jackson Laboratory launch rare disease gene therapy collaboration
The three institutions have announced a joint initiative to develop gene therapies targeting rare diseases, pooling expertise across genomics, clinical research, and model-organism biology.
The Broad Institute of MIT and Harvard, Boston Children's Hospital, and the Jackson Laboratory in Bar Harbor, Maine, have announced a new collaboration — described as the Center for Therapeutic Genetics — aimed at accelerating the development of gene therapies for rare diseases.
The partnership is intended to combine the Broad Institute's genomics and genome-editing infrastructure with Boston Children's Hospital's paediatric clinical and translational research capabilities and the Jackson Laboratory's long-standing expertise in mouse models and mammalian genetics. Stat News reported the announcement on 21 July 2026; further details about which disease areas or gene targets will be prioritised in the first instance had not been disclosed at time of publication.
Rare-disease gene therapy has attracted substantial institutional and public funding in recent years. The ARPA-H agency committed $160 million to bespoke gene-editing treatments for rare diseases in July 2026, and the broader field continues to face challenges including high per-patient development costs, small trial populations, and manufacturing scalability. The new initiative appears designed to share those development burdens across institutions with complementary strengths.
Full programmatic details, including disease targets, funding sources, and governance arrangements, are expected to follow the initial announcement.
Plain-language version
For patients, families, and general readers. Educational only — not medical advice.
Three major US research organisations — the Broad Institute, Boston Children's Hospital, and the Jackson Laboratory — have announced they will work together to develop gene therapies for rare diseases. Gene therapies are experimental treatments that aim to correct or replace faulty genes that cause disease.
Rare diseases often affect small numbers of people, which can make it difficult for any single organisation to develop treatments on its own. By combining their different areas of expertise — from studying the genome, to working with patients in hospital, to studying how genes work in animals — the three institutions hope to make progress more quickly.
No specific diseases or treatments have been named yet; the announcement describes the partnership's aims rather than a finished programme. Researchers and families affected by rare diseases may see further details emerge as the collaboration publishes its plans.
This is an educational summary, not medical advice. If anything here raises questions for you, please speak with your GP or a clinical professional.
Sources
Read the original reporting — these are the public sources this summary draws from.
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Primary source Stat News · 2026-07-21STAT+: Broad Institute, partners unveil new initiative to develop gene therapies for rare diseases